Broad Institute Gets Patent on Revolutionary Gene-Editing Method

April 16, 2014

Broad Institute Gets Patent on Revolutionary Gene-Editing Method

The Harvard-MIT genomic science institute stays mute on how it will assert control over the tools expected to speed cures and change gene therapy.

One of the most important genetic technologies developed in recent years is now patented, and researchers are wondering what they will and won’t be allowed to do with the powerful method for editing the genome.

On Tuesday, the Broad Institute of MIT and Harvard announced that it had been granted a patent covering the components and methodology for CRISPR—a new way of making precise, targeted changes to the genome of a cell or an organism. CRISPR could revolutionize biomedical research by giving scientists a more efficient way of re-creating disease-related mutations in lab animals and cultured cells; it may also yield an unprecedented way of treating disease (see “Genome Surgery”).

The patent, issued just six months after its application was filed, covers a modified version of the CRISPR-Cas9 system found naturally in bacteria, which microbes use to defend themselves against viruses. The patent also covers methods for designing and using CRISPR’s molecular components.

The patent describes how the tools could be used to treat diseases, and lists many specific conditions from epilepsy, to Huntington’s, to autism, and macular degeneration. One of the most exciting possibilities for CRISPR is its potential to treat genetic disorders by directly correcting mutations on a patient’s chromosomes. That would enable doctors to treat diseases that cannot be addressed by more traditional methods, a goal already set by a startup cofounded by Zhang called Editas Medicine (see “New Genome-Editing Method Could Make Gene Therapy More Precise and Effective”).

Another founder of Editas, Jennifer Doudna, and her institute, the University of California, have a pending patent application for CRISPR technology. How that west coast application will be affected is not yet clear. It’s also unclear what impact the Broad’s claims on the technology will have on its commercial use and on basic research.

Chelsea Loughran, an intellectual property litigation lawyer who has been following CRISPR over the last year, says that lots of people are already using CRISPR and it’s not clear if it will now become harder for them to do that. “All of that is in the hands of MIT and the Broad,” she says.

While MIT, Harvard, and the Broad all jointly own the CRISPR patents announced yesterday, the Broad’s technology licensing office is managing decisions about who will get licenses to use the technology, says Lita Nelsen, director of the MIT Technology Licensing Office. (Licenses are formal permissions to use a patented technology, often in exchange for money.)

A spokesperson for the Broad says that “specific details around licensing aren’t available at this time, but the Broad does intend to make this technology broadly available to scientists.”

Nelsen says MIT’s experience with patenting another genetic technology—short interfering RNAs— provides a good example of what to expect for the CRISPR patents. Short interfering RNAs are a family of molecules that can be used to silence genes in many organisms. Those molecules are widely used by basic research scientists.

Nelsen’s office granted licenses to use the interfering RNA technology to different groups, with different sorts of permission. A startup called Alnylam got an exclusive license to use the technology to try to develop treatments for patients (see “The Therapeutic Potential of RNAi”). In addition, MIT granted non-exclusive licenses to a few companies that wanted to sell the molecular components of the gene-silencing technique.

The reason universities patent basic research tools is to provide the incentive to develop it, says Nelsen. Transforming such a technology into a medical treatment is an immensely expensive process, she says. “If you didn’t patent, that wouldn’t get done,” says Nelsen.

MIT does not go after academic scientists who produce lab-made versions of the molecular components for the gene-silencing technology, she says. If the Broad technology licensing office follows that example, then the new patents should not affect academics wanting to use the tool for basic research, says Nelsen. That would be good news, because CRISPR is thought to be a huge boon for biological science. For now, however, the Broad is keeping a tight lid on their plans for the patent.